Table 1.
Baseline patient characteristics and disease variables
| Total (N = 15) | Cohort 1 (n = 5) | Cohort 2 (n = 3) | Cohort 3 (n = 4) | Cohort 4 (n = 3) | |
|---|---|---|---|---|---|
| Age, median y, (range) | 50 (20-68) | 48 (26-68) | 56 (20-58) | 53 (32-68) | 41 (27-50) |
| Sex, n (%) | |||||
| Male | 12 (80) | 4 (80) | 3 (100) | 3 (75) | 2 (67) |
| Female | 3 (20) | 1 (20) | 0 | 1 (25) | 1 (33) |
| ECOG PS, n (%) | |||||
| 0 | 2 (13) | 0 | 0 | 1 (25) | 1 (33) |
| 1 | 11 (73) | 5 (100) | 1 (33) | 3 (75) | 2 (67) |
| 2 | 2 (13) | 0 | 2 (67) | 0 | 0 |
| ELN risk, n (%) | |||||
| Favorable | 1 (7) | 0 | 0 | 0 | 1 (33) |
| Intermediate* | 8 (53) | 2 (40) | 3 (100) | 2 (50) | 1 (33) |
| Adverse | 6 (40) | 3 (60) | 0 | 2 (50) | 1 (33) |
| WBC, ×103/µL, median (range) | 2.9 (0.7-23.0) | 2.9 (1.8-6.2) | 7.5 (3.6-23.0) | 2.4 (0.7-3.7) | 2.1 (1.6-6.4) |
| PB blasts, %, median (range) | 10 (0-78) | 19 (0-32) | 28 (0-78) | 5 (0-33) | 0 (0-0) |
| BM blasts, %, median (range) | 40 (0-91) | 50 (5-80) | 75 (5-80) | 53 (10-91) | 0 (0-40)† |
| Lines of therapy, median (range) | 3 (1-5) | 2 (2-3) | 3 (2-3) | 3 (1-4) | 4 (3-5) |
| Prior allogeneic HSCTs, n (%) | |||||
| 1 | 9 (60) | 3 (60) | 2 (67) | 3 (75) | 1 (33) |
| 2 | 4 (27) | 1 (20) | 1 (33) | 0 | 2 (67) |
| 3 | 2 (13) | 1 (20) | 0 | 1 (25) | 0 |
| Donor type, n (%) | |||||
| MUD | 8 (53) | 3 (60) | 2 (67) | 1 (25) | 2 (67) |
| Matched related | 1 (7) | 1 (20) | 0 | 0 | 0 |
| MMUD | 4 (27) | 0 | 1 (33) | 2 (50) | 1 (33) |
| Haploidentical related | 2 (13) | 1 (20) | 0 | 1 (25) | 0 |
| Days since prior HSCT, median (range) | 154 (63-904) | 133 (68-292) | 154 (63-533) | 278 (96-904) | 472 (94-860) |
ECOG, Eastern Cooperative Oncology Group; ELN, European LeukemiaNet; PS, performance status; WBC, white blood cell.
*
According to ELN 2010 definitions, intermediate-I and -II categories were merged.
†
Cohort 4 included 2 patients with extramedullary AML.